TABLE 1.
Baseline characteristics of the study participants
| Characteristics | Safety study (N = 171) | Immune response study (N = 53) |
|---|---|---|
| Sex: male, n (%) | 128 (74.9%) | 38 (71.7%) |
| Age (years), median (range) | 46.0 (20‐89) | 41.7 (20‐78) |
| Body weight (kg), median (range) | 42.3 (18‐81) | 38.6 (18‐81) |
| MD type, n (%) | ||
| Duchenne/Becker MD | 70 (40.9%) | 24 (45.3%) |
| Myotonic dystrophy type 1 | 72 (42.1%) | 20 (37.7%) |
| Fukuyama congenital MD | 7 (4.1%) | 4 (7.5%) |
| Facioscapulohumeral MD | 5 (2.8%) | 1 (1.9%) |
| Limb‐girdle MD | 10 (5.8%) | 1 (1.9%) |
| Other | 7 (4.1%) | 3 (5.7%) |
| Underlying disease, n (%) | ||
| Heart failure | 89 (52.0%) | 29 (54.7%) |
| Hypertension | 7 (4.1%) | 0 |
| Diabetes mellitus | 19 (11.1%) | 2 (3.8%) |
| Hyperlipidemia | 23 (13.5%) | 3 (5.7%) |
| Any allergic disease a | 5 (2.9%) | 3 (5.7%) |
| Ambulatory status, n (%) | ||
| Ambulatory | 5 (2.9%) | 2 (3.8%) |
| Wheelchair user | 80 (46.8%) | 10 (18.8%) |
| Bedridden | 86 (50.3%) | 41 (77.4%) |
| Respiratory status, n (%) | ||
| None | 30 (17.5%) | 2 (3.8%) |
| NPPV | 77 (45.0%) | 23 (43.4%) |
| TPPV | 64 (37.4%) | 28 (52.8%) |
|
Did he/she need informed consent by proxy (parents or siblings)? | ||
| Yes, n (%) | 39 (22.8%) | 16 (30.2%) |
Abbreviations: MD, muscular dystrophy; NPPV, noninvasive positive pressure ventilation; TPPV, tracheostomy positive pressure ventilation.
a
Any allergic diseases include atopic dermatitis and asthma.